To one day cure type 1 diabetes (T1D), we must halt the destruction of beta cells that produce insulin, and we need to make sure that the person then has enough of those cells. Until that day, JDRF funds prevention research that focuses on the former. At the American Diabetes Association’s 78th Scientific Sessions, JDRF-supported researcher Michael Haller, MD, MS-CI, a primary investigator and the division chief of pediatric endocrinology at the University of Florida, presented new research results from the Diabetes TrialNet ATG-GCSF New Onset Study that suggest a potential path to preserve beta cell function in people with new onset T1D.

The study looked at whether a low-dose of the drug known as anti-thymocyte globulin, trade name Thyroglobulin (ATG), used alone or in combination with pegylated granulocyte-colony stimulating factor, trade name Neulasta (GCSF), a drug commonly used to increase white blood cell counts in patients receiving chemotherapy helps people newly diagnosed with T1D maintain insulin production.

The Diabetes TrialNet ATG-GCSF New Onset Study found that at one year after the start of treatment, low-dose ATG preserved beta cell function and improved insulin production. Additionally, hemoglobin A1c (HbA1c) levels were significantly lower in people treated with low-dose ATG alone and in people treated with low-dose ATG combined with GCSF. Low-dose ATG combined with GCSF, however, did not enhance beta cell preservation.

“The results of treating those newly diagnosed with low-dose ATG are encouraging and provide new hope for people early in their diagnosis of type 1 diabetes,” said Dr. Haller, primary investigator and the division chief of pediatric endocrinology at the University of Florida. “Rarely have we found agents capable of both preserving insulin producing beta cells and reducing A1c, and I am eager to design additional studies on the role of ATG in preventing the onset of the disease.”

The findings demonstrate that a single course of low-dose ATG, an FDA approved agent with several decades of use in transplant medicine, can preserve insulin-producing beta cells and reduce HbA1c for at least one year in newly diagnosed patients.

The study included 89 participants between the ages of 12 and 45, who were tested within 100 days of being diagnosed with T1D. Participants were divided into three groups: one group received ATG, one group received ATG combined with GCSF, and one group received a placebo. In the treatment phase, all participants had two days of IV infusions, then six injections over a 10-week period. They were then tested every three to six months for the next 21 months.

These data are the results after one year of study; the trial will be completed when all participants complete two years of study visits. Final results are expected in 2019. The “ATG-GCSF New Onset Study” builds on prior findings of an initial pilot study that suggested ATG combined with GCSF preserved insulin production for more than a year after treatment in people who had T1D for less than two years.